Marisa Wexler, MS,  senior science writer—

The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation and rare pediatric disease designation to BBDF-101, an investigational treatment for Batten disease. Theranexus is developing BBDF-101 — which seeks to slow the progression of the inherited neurological condition — pursuant to…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a gene therapy being developed by Neurogene for the treatment of CLN7 Batten disease. This marks the second Neurogene gene therapy for Batten disease to receive orphan drug designation from the FDA this summer.

The number of fluid-containing cellular compartments called vacuoles within immune cells could help to predict the severity of Batten disease, a new study suggests. The study, “Quantifying lymphocyte vacuolization serves as a measure of CLN3 disease severity,” was published in JIMD Reports. Vacuoles are compartments…

Two previously-unknown mutations in the CLN5 gene were reported to cause CLN5 disease, a form of late infantile Batten disease, in a new study. The study also represents the second-ever publication describing CLN5 disease in people in Pakistan, hinting at a more worldwide distribution of the condition than…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Amicus Therapeutics‘ AT-GTX-502, an investigational gene therapy for Batten disease caused by mutations in the CLN3 gene, the company announced in a press release. This designation is given to investigational therapies that…

Polaryx Therapeutics has received approval from the U.S. Food and Drug Administration (FDA) to open a clinical trial into PLX-200 as a potential treatment for juvenile Batten disease. Juvenile Batten, also known as Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) or CLN3 disease, is caused by mutations in…

An investigational gene therapy for late infantile (CLN2) Batten disease showed a good safety profile in a small clinical trial. The gene therapy also slowed the progression of the disease, though not to the extent of current standard-of-care treatment. Researchers are now working on more effective ways to deliver…